dc.contributor.author |
Torre Fornell, Rafael de la |
dc.contributor.author |
De Sola Llopis, Susana |
dc.contributor.author |
Xicota Vila, Laura, 1987- |
dc.contributor.author |
Cuenca Royo, Aida Ma, 1981- |
dc.contributor.author |
Rodriguez, Joan |
dc.contributor.author |
Gomis González, Maria, 1988- |
dc.contributor.author |
Hernández, Gimena |
dc.contributor.author |
Del Hoyo Soriano, Laura |
dc.contributor.author |
Ozaita Mintegui, Andrés, 1969- |
dc.contributor.author |
Dierssen, Mara |
dc.contributor.author |
TESXF Study Group |
dc.contributor.author |
Principe, Alessandro |
dc.contributor.author |
Espadaler, Josep María |
dc.contributor.author |
Roca, Laia |
dc.contributor.author |
Del Hoyo Soriano, Laura |
dc.contributor.author |
Farré Albaladejo, Magí |
dc.contributor.author |
Fitó Colomer, Montserrat |
dc.contributor.author |
León Jorba, Alba |
dc.contributor.author |
Banea, Ovideo |
dc.contributor.author |
Maldonado, Rafael, 1961- |
dc.contributor.author |
Busquets Garcia, Arnau, 1985- |
dc.date.accessioned |
2019-04-30T07:50:39Z |
dc.date.issued |
2020 |
dc.identifier.citation |
de la Torre R, de Sola S, Farré M, Xicota L, Cuenca-Royo A, Rodriguez J et al. A phase 1, randomized double-blind, placebo controlled trial to evaluate safety and efficacy of epigallocatechin-3-gallate and cognitive training in adults with Fragile X syndrome. Clin Nutr. 2020;39(2):378-87. DOI 10.1016/j.clnu.2019.02.028 |
dc.identifier.issn |
0261-5614 |
dc.identifier.uri |
http://hdl.handle.net/10230/37158 |
dc.description.abstract |
Background & aims: Despite the wide spectrum of experimental compounds tested in clinical trials, there is still no proven pharmacological treatment available for Fragile-X syndrome (FXS), since several targeted clinical trials with high expectations of success have failed to demonstrate significant improvements. Here we tested epigallocatechin-3-gallate (EGCG) as a treatment option for ameliorating core cognitive and behavioral features in FXS. Methods: We conducted preclinical studies in Fmr1 knockout mice (Fmr1−/y) using novel object-recognition memory paradigm upon acute EGCG (10 mg/kg) administration. Furthermore we conducted a double-blind placebo-controlled phase I clinical trial (TESXF; NCT01855971). Twenty-seven subjects with FXS (18–55 years) were administered of EGCG (5–7 mg/kg/day) combined with cognitive training (CT) during 3 months with 3 months of follow-up after treatment discontinuation. Results: Preclinical studies showed an improvement in memory using the Novel Object Recognition paradigm. We found that FXS patients receiving EGCG + CT significantly improved cognition (visual episodic memory) and functional competence (ABAS II-Home Living skills) in everyday life compared to subjects receiving Placebo + CT. Conclusions: Phase 2 clinical trials in larger groups of subjects are necessary to establish the therapeutic potential of EGCG for the improvement of cognition and daily life competences in FXS. |
dc.description.sponsorship |
This work was supported by the Spanish Ministry of Economy and Competitiveness; ‘Centro de Excelencia Severo Ochoa 2013-2017’; the ‘Centres de Recerca de Catalunya’ Programme / Generalitat de Catalunya’; the Centro de Investigación Biomédica en Red Enfermedades Raras (CIBER) of Rare Diseases; DIUE de la Generalitat de Catalunya [Grups consolidats SGR 2014/1125]; Ministerio de Economía, Industria y Competitividad de España MINECO [SAF2013-49129-C2-1-R and SAF2016-79956-R]. |
dc.format.mimetype |
application/pdf |
dc.language.iso |
eng |
dc.publisher |
Elsevier |
dc.relation.ispartof |
Clinical Nutrition. 2020;39(2):378-87 |
dc.rights |
© Elsevier http://dx.doi.org/10.1016/j.clnu.2019.02.028 |
dc.title |
A phase 1, randomized double-blind, placebo controlled trial to evaluate safety and efficacy of epigallocatechin-3-gallate and cognitive training in adults with Fragile X syndrome |
dc.type |
info:eu-repo/semantics/article |
dc.identifier.doi |
http://dx.doi.org/10.1016/j.clnu.2019.02.028 |
dc.subject.keyword |
Fragile-X syndrome |
dc.subject.keyword |
Epigallocatechin gallate |
dc.subject.keyword |
Cognition |
dc.subject.keyword |
Functionality |
dc.relation.projectID |
info:eu-repo/grantAgreement/ES/1PE/SAF2013-49129-C2-1-R |
dc.relation.projectID |
info:eu-repo/grantAgreement/ES/1PE/SAF2016-79956-R |
dc.rights.accessRights |
info:eu-repo/semantics/openAccess |
dc.type.version |
info:eu-repo/semantics/acceptedVersion |